A New Milestone For ISIS-SMN Rx

Isis Pharmaceuticals announced last week that the first child in the 12 mg group was dosed in the ongoing Phase 2 study evaluating ISIS-SMN_Rx in children with spinal muscular atrophy (SMA). Isis also announced that the first child was dosed in an open–label extension study, which is being offered to those children with SMA who have completed dosing in Isis' previous studies. Furthermore dosing in the Phase 2 study in infants with SMA is being extended.  Under the extension, infants who have completed the three initially scheduled study doses will be eligible to receive an additional dose of 12 mg. Isis also announced that the first infant in the study has received a fourth dose of ISIS-SMN_Rx approximately six months after the three initial doses were completed. 

"The progress we and Biogen Idec have made on ISIS-SMNRx exemplifies the value of our strategic alliance and the benefit of working closely together with combined expertise to support rapid development.   We also benefit financially.  Including the more than $9 million announced today, we will have received more than $45 million in upfront fees, milestone and other payments from Biogen Idec as ISIS-SMNRx has advanced.  This year we anticipate making significant progress in this and other programs we are working on with the Biogen Idec team," said B. Lynne Parshall, chief operating officer at Isis.  "The ongoing multiple-dose studies in infants with Type I SMA and in children with Type II and Type III SMA are progressing, and we look forward to sharing the top-line data from these studies this quarter and more detailed results at the American Academy of Neurology meeting in April."